NCT06351462

Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients

• Status: Not Yet Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 220 (estimated)
• Sponsor: Assistance Publique - Hôpitaux de Paris · Academic / Other
• Sex: All
• Age: 15 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

The long term burden of morbidity and mortality in the natural history of sickle cell disease has not been compared up to date to the risks and mortality of a curative option like bone marrow transplantation in severe sickle-cell disease patients. Given this lack of data, primary-care Sickle Cell Disease (SCD) physicians and transplant physicians are prevented from a factual debate over the benefit/risk ratio for each patient and refining indications of transplant in patients. Therefore, the present study seeks to describe and compare the very long-term outcomes after either Human Leukocyte Antigen (HLA) -matched sibling transplantation (study arm) and "non-transplant care" for severe sickle cell disease SCA patients in order to yield robust comparative data regarding both arms. The main objective is to assess the benefit of Hematopoietic stem cell transplantation (HSCT) regarding quality of life compared to standard care after 10 years, in patients with severe Sickle Cell Disease (SCD).

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
OTHER	Spermogram	Spermogram will be proposed to men
OTHER	Hospital Anxiety and Depression Scale (HADS)	Anxiety and depression will be evaluated 10 years after HSCT
OTHER	SF36 Quality of life questionnaire	Quality of life will be evaluated 10 years after HSCT
OTHER	Psychologist interview	During a follow-up visit
OTHER	Optional sera banking	One in the study
OTHER	Optional DNA banking	Once in the study

Timeline

• Start date: 2024-05-01
• Primary completion: 2027-05-01
• Completion: 2027-05-01
• First posted: 2024-04-08
• Last updated: 2024-04-19